Oral Drug OK'd for Bile Acid Synthesis Disorders

Cholic acid capsules become first approved treatment for these genetic conditions.

by John Gever John Gever,Managing Editor, MedPage Today
March 18, 2015

WASHINGTON -- The FDA approved cholic acid capsules (Cholbam) to treat a group of bile acid synthesis disorders arising from single enzyme defects, the agency announced Tuesday.

Also covered in the approval are so-called peroxisomal disorders including Zellweger spectrum disorders.

Collectively, these are rare disorders in which genetic mutations leave people unable to synthesize cholic acid. In such patients, bile acid intermediates accumulate to toxic levels in the liver, and fats and fat-soluble vitamins cannot be metabolized properly. Children typically fail to thrive.

Cholbam consequently serves as a replacement therapy. The approval covers children as young as 3 weeks as well as adults, the FDA said.

Its efficacy in bile acid synthesis disorders was demonstrated in an open-label, 50-patient trial, mostly young children at enrollment, with body weight and liver function test results as the main outcomes. Positive responses were seen in 64%, the agency said, with a 3-year survival rate above 60%.

For peroxisomal disorders, the drug was tested in 29 patients, among whom 46% showed favorable effects on weight and liver function. The 3-year survival rate was 42%.

The drug's efficacy was limited, however -- neurologic and other manifestations were not improved with treatment, the FDA noted.

Diarrhea was the most common side effect seen in the trials.

As a reward for bringing a drug for rare childhood conditions through the approval process, the FDA is giving Cholbam's developer, Baltimore-based Asklepion Pharmaceuticals, a "pediatric disease priority review voucher" -- entitling it to receive fast-track review for another product that otherwise would be ineligible for it.

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